Author Archives: admin


Stem Cell Clinic > Articles by: admin

Ixazomib Extends Survival in Multiple Myeloma for Patients Not Treated With Stem Cell Transplantation – Pharmacy Times

By Stem Cell Treatment

Ixazomib Extends Survival in Multiple Myeloma for Patients Not Treated With Stem Cell Transplantation

The phase 3 TOURMALINE-MM4 study evaluated single-agent oral ixazomib as first-line maintenance therapy versus placebo in 706 adult patients diagnosed with MM not treated with stem cell transplantation, who have completed 6 to 12 months of initial therapy and achieved a partial response or better.

New data from the study have demonstrated statistically significant improvement in PFS, meeting the primary endpoint of the trial, Takeda announced. According to the press release, this is the first industry-sponsored phase 3 trial to investigate the concept of switch maintenance, the use of medications not included in initial induction therapy, in this setting.

First approved by the FDA in November 2015, ixazomib is an oral proteasome inhibitor being studied across several MM treatment settings, according to Takeda. It is currently indicated in combination with lenalidomide and dexamethasone for the treatment of patients with MM who have received at least 1 prior therapy.

The TOURMALINE clinical development program includes ongoing clinical trials involving ixazomib in MM:

Reference

Phase 3 Trial Ninlaro (ixazomib) as First Line Maintenance Therapy Met Primary Endpoint in Multiple Myeloma Patients not treated with Stem Cell Transplantation [news release]. Takedas website. https://www.takeda.com/newsroom/newsreleases/2019/phase-3-trial-of-ninlaro-ixazomib-as-first-line-maintenance-therapy-met-primary-endpoint-in-multiple-myeloma-patients-not-treated-with-stem-cell-transplantation/. Accessed November 7, 2019.

Read more here:
Ixazomib Extends Survival in Multiple Myeloma for Patients Not Treated With Stem Cell Transplantation - Pharmacy Times

Phase III trial of Ninlaro as first-line maintenance therapy meets PFS in multiple myeloma – The Cancer Letter

By Stem Cell Treatment

publication date: Nov. 8, 2019

The phase III Takeda TOURMALINE-MM4 study met its primary endpoint of progression-free survival in multiple myeloma not treated with stem cell transplantation.

The trial evaluated the effect of single-agent oral Ninlaro (ixazomib) as a first-line maintenance therapy versus placebo, and demonstrated statistically significant improvement in PFS, according to Takeda. TOURMALINE-MM4 is the first industry-sponsored phase III trial to explore the concept of switch maintenance, the use of medicines not included in initial induction therapy, in this setting.

Ninlaro is not approved for this specific use.

TOURMALINE-MM4 is a randomized, placebo-controlled, double-blind phase III study of 706 patients who have completed 6-12 months of initial therapy and achieved a partial response or better.

Inovio demonstrates 80% 6-month PFS in phase II glioblastoma multiforme study with INO-5401 + Libtayo

A phase II study (NCT03491683) of newly diagnosed glioblastoma multiforme combining Inovios INO-5401 and INO-9012 in combination with Libtayo (cemiplimab) showed that 80% (16 of 20) of MGMT gene promoter methylated patients and 75% (24 of 32) of unmethylated patients were progression-free at six months measured from the time of their first dose (n = 52), substantially exceeding historical standard-of-care data.

INO-5401 is a T cell-activating immunotherapy encoding for three tumor-associated antigens (hTERT, WT1, and PSMA). INO-9012 is an immune activator encoding IL-12, and Libtayo is a PD-1 blocking antibody developed by Regeneron Pharmaceuticals in collaboration with Sanofi.

The data were to be presented at the Society for Immunotherapy of Cancer 2019 Annual Meeting in National Harbor, MD, Nov. 6-10.

This immunotherapy combination with a Continue reading Phase III trial of Ninlaro as first-line maintenance therapy meets PFS in multiple myelomaTo access this members-only content, please log in.Institutional subscribers, please log in with your IP.If you're not a subscriber why not join today?To gain access to the members only content click here to subscribe.You will be given immediate access to premium content on the site.Click here to join.

See more here:
Phase III trial of Ninlaro as first-line maintenance therapy meets PFS in multiple myeloma - The Cancer Letter

AgeX Therapeutics to Present at Investing in the Age of Longevity Conference – Business Wire

By Stem Cell Treatment

ALAMEDA, Calif.--(BUSINESS WIRE)--AgeX Therapeutics, Inc. (AgeX; NYSE American: AGE), a biotechnology company focused on developing therapeutics for human aging and regeneration, announced today that the company will present at Investing in the Age of Longevity, organized by Master Investor, Ltd., on Wednesday, November 13 at Science Gallery London.

Details of the companys participation:

The full event program is available here.

About AgeX Therapeutics

AgeX Therapeutics, Inc. (NYSE American: AGE) is focused on developing and commercializing innovative therapeutics for human aging. Its PureStem and UniverCyte manufacturing and immunotolerance technologies are designed to work together to generate highly-defined, universal, allogeneic, off-the-shelf pluripotent stem cell-derived young cells of any type for application in a variety of diseases with a high unmet medical need. AgeX has two preclinical cell therapy programs: AGEX-VASC1 (vascular progenitor cells) for tissue ischemia and AGEX-BAT1 (brown fat cells) for Type II diabetes. AgeXs revolutionary longevity platform induced Tissue Regeneration (iTR) aims to unlock cellular immortality and regenerative capacity to reverse age-related changes within tissues. AGEX-iTR1547 is an iTR-based formulation in preclinical development. HyStem is AgeXs delivery technology to stably engraft PureStem cell therapies in the body. AgeX is developing its core product pipeline for use in the clinic to extend human healthspan and is seeking opportunities to establish licensing and collaboration agreements around its broad IP estate and proprietary technology platforms.

For more information, please visit http://www.agexinc.com or connect with the company on Twitter, LinkedIn, Facebook, and YouTube.

Forward-Looking Statements

Certain statements contained in this release are forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Any statements that are not historical fact including, but not limited to statements that contain words such as will, believes, plans, anticipates, expects, estimates should also be considered forward-looking statements. Forward-looking statements involve risks and uncertainties. Actual results may differ materially from the results anticipated in these forward-looking statements and as such should be evaluated together with the many uncertainties that affect the business of AgeX Therapeutics, Inc. and its subsidiaries, particularly those mentioned in the cautionary statements found in more detail in the Risk Factors section of AgeXs Annual Report on Form 10-K and Quarterly Reports on Form 10-Q filed with the Securities and Exchange Commissions (copies of which may be obtained at http://www.sec.gov). Subsequent events and developments may cause these forward-looking statements to change. AgeX specifically disclaims any obligation or intention to update or revise these forward-looking statements as a result of changed events or circumstances that occur after the date of this release, except as required by applicable law.

Read the original here:
AgeX Therapeutics to Present at Investing in the Age of Longevity Conference - Business Wire

Tears And Joy This Toddler with Down Syndrome Who Was Battling Leukemia Is Finally Cancer Free – SurvivorNet

By Stem Cell Treatment

Proud auntie Paola Mayfield says her niece, who has Down syndrome, is in remission from the leukemia that she battled for two years.

Last year I went to Colombia when I was pregnant, I needed to see my sister and my niece, Mayfield wrote alongside a photo of niece standing on a hospital bed with her arm attached to some medical devices. My niece has Down Syndrome and had been battling leukemia for about 2 years. But TODAY we received the greatest news! She is finally cancer free!

Paola said that the journey has been extremely difficult. With tears in my eyes I feel full of joy and happiness because I know how hard it has been the past few years for my sister.

And she wanted others who are struggling and fighting for their lives to know that there is always hope: For those who battle everyday to have another day of life, stay strong and dont lose faith. Thank you [hearts].

Leukemia is the most common type of childhood cancer. The most frequent type of childhood leukemia is acute lymphoblastic leukemia (ALL). Three out every four cases of childhood leukemia are diagnosed as acute, meaning that the leukemia can progress quickly, and if not treated, would probably be fatal within a few months.

Acute lymphoblastic leukemia is a rare cancer thatoccurs when the bone marrow makes too much of a type of white blood cell calledlymphocytes, according to the National Cancer Institute. Signs of childhood ALL include fever and bruising. The disease can be detected using tests that examine the blood and bone marrow. Over time, there has been a lot of improvement in treatments for childhood leukemia.

There are several different approaches to treating the disease, and the treatment plan will depend on the type of ALL. Chemotherapy, radiation, chemotherapy with a stem cell transplant, and targeted therapy are all considered standard treatment, according to the American Cancer Society.

The next most common type of childhood leukemia is called acute myeloid leukemia, which occurs when the bone marrow makes a large number of abnormal blood cells called myeloblasts. As these cells build up, they prevent the growth ofhealthy white blood cells, red blood cells, and platelets.

Learn more about SurvivorNet's rigorous medical review process.

Proud auntie Paola Mayfield says her niece, who has Down syndrome, is in remission from the leukemia that she battled for two years.

Last year I went to Colombia when I was pregnant, I needed to see my sister and my niece, Mayfield wrote alongside a photo of niece standing on a hospital bed with her arm attached to some medical devices. My niece has Down Syndrome and had been battling leukemia for about 2 years. But TODAY we received the greatest news! She is finally cancer free!

Paola said that the journey has been extremely difficult. With tears in my eyes I feel full of joy and happiness because I know how hard it has been the past few years for my sister.

And she wanted others who are struggling and fighting for their lives to know that there is always hope: For those who battle everyday to have another day of life, stay strong and dont lose faith. Thank you [hearts].

Leukemia is the most common type of childhood cancer. The most frequent type of childhood leukemia is acute lymphoblastic leukemia (ALL). Three out every four cases of childhood leukemia are diagnosed as acute, meaning that the leukemia can progress quickly, and if not treated, would probably be fatal within a few months.

Acute lymphoblastic leukemia is a rare cancer thatoccurs when the bone marrow makes too much of a type of white blood cell calledlymphocytes, according to the National Cancer Institute. Signs of childhood ALL include fever and bruising. The disease can be detected using tests that examine the blood and bone marrow. Over time, there has been a lot of improvement in treatments for childhood leukemia.

There are several different approaches to treating the disease, and the treatment plan will depend on the type of ALL. Chemotherapy, radiation, chemotherapy with a stem cell transplant, and targeted therapy are all considered standard treatment, according to the American Cancer Society.

The next most common type of childhood leukemia is called acute myeloid leukemia, which occurs when the bone marrow makes a large number of abnormal blood cells called myeloblasts. As these cells build up, they prevent the growth ofhealthy white blood cells, red blood cells, and platelets.

Learn more about SurvivorNet's rigorous medical review process.

Visit link:
Tears And Joy This Toddler with Down Syndrome Who Was Battling Leukemia Is Finally Cancer Free - SurvivorNet

Tales of medical misinformation and misadventures – Stuff.co.nz

By Stem Cell Treatment

There's an old saying that "if you have your health, then you have everything".

Modern healthcare and society's approach to diseaseare covered in some new podcasts that are worth taking a deep diveinto.

Popculture podcast Endless Threadfinds weekly subject matter by diving into the enormousonline communities which congregate on Reddit. Presenters Ben Brock Johnson and Amory Sivertsonhave released a special five-part series called Infectious, which focuses on recent measles epidemics and the rise of the anti-vaccination movement.

At the centre of this story is the internet and the ways in which misinformation about vaccines and mistrust of medicines has been spread. This is a well-researched documentary series featuring medical experts, public health officials and parents and seeks to untangle fact from fiction.

Ben Brock Johnson, left, and Amory Sivertson, right, host the Endless Thread podcast, which dives into the world of vaccines and anti-vaxxers.

READ MORE:* Cryptoqueens and scam goddesses: Exploring fraud via podcasts* Podcasts that are an ode to nature* Why you need to listen to Dolly Parton's America

ENDLESS THREAD

The Endless Thread podcast delves into Reddit's vast communities to explore some of the most compelling stories the Internet has to offer.

Last Dayalso tackles an epidemic but in this case it is the United States' opioid crisis as seen through the lenses of family members who have lost a relative to an opioid overdose. The first episode charts the last day of life of presenterStephanie Wittels Wachs' brother, Harris Wittels, who died of an overdose at the age of 30 in 2015.

He was a comedian and TV writer who wrote for The Sarah SilvermanShow and Aziz Ansari's Master of None. This series provides a deeply personal look at the disease of addiction and how we as a society treat those suffering from it.

Stem cell treatment has been touted as a miracle option for pain and healing in patients and the latest Wondery podcast examines what happens when patients in Texas get a bad batch of stem cells.

Presented by medical journalist, Laura Biel, who also presented the hugely popular Dr Death podcast, Bad Batch exposes greed and desperation in this highly unregulated multi-million dollar industry.

Here is the original post:
Tales of medical misinformation and misadventures - Stuff.co.nz

‘My shock cancer scare and the anonymous German who saved me’ – Birmingham Live

By Stem Cell Treatment

Sam Williams was just a normal student attending the University of Birmingham before his life was torn apart in an instant.

A week before sitting his final exams he was struck ill with glandular fever - and then contracted sepsis in hospital.

Yet tests would reveal further news that would change Sam's life forever.

Doctors discovered he had a life threatening rare blood cancer named Myelodysplastic syndrome, otherwise known as MDS.

But over the next two years he beat the odds by fighting the cancer - with the help of an anonymous German stem cell donor.

Sam, from Walsall, was first diagnosed with the condition aplastic anemia aged just three years old. The drug treatment he received was successful and he lived a normal life growing up.

But after turning 21, with the end of university in sight, his life was changed forever after being hospitalised.

"It was completely sudden," recalls Sam, now aged 23.

"It was found that I didn't have aplastic anemia anymore, it was a condition called MDS. That was a life threatening condition.

"That was a real shock. I was healthy for so many years. To suddenly be told you have a serious blood disorder and need a blood transplant, that was a massive shock for me."

Doctors told Sam he would need a stem cell donor. It took six weeks of waiting before he found one through leading blood charity Anthony Nolan.

"I had to go back to the hospital every week, and every week they were telling me 'sorry we haven't found anybody' and that felt like it was going to go on forever," he recalled.

"Literally whilst I was talking to the consultant an email popped up on his screen saying that a donor was found for me. That came as a real shock."

Video Unavailable

Click to playTap to play

Play now

After Sam received the life saving donation, he felt compelled to reach out to the man who gave him a second chance at life.

However, there are strict guidelines surrounding contact between transplant recipients and donors.

Anonymity is paramount and any information passed between the two parties is discussed and organised by the Specialist Nurse in Organ Donation (SN-OD), the Recipient Transplant Coordinator and the Donor Records Department (DRD).

"For me it would have been almost certain that I wouldn't be alive today if he had not donated," says Sam.

"Shortly after the transplant, I wrote him a letter.

"I was trying to explain at the time that I was extremely thankful for what he has done but it was impossible to put it into words - thank you just wasn't enough.

"I [wrote] how difficult it was to put into words how to thank them for what they had done, because they had effectively given me a second chance at life which was of course something very significant to me.

"I wasn't able to say much more than that due to data protection regulations. I could say that I was an adult, for example, but I couldn't give any details of my age, where I lived, etc."

"I wrote just a very short letter, it took me three days of redrafting that before I got the wording correct. I just graduated from university at the time, I found that letter harder to write than my dissertation.

"Unfortunately [he] didn't respond. I would have been absolutely over the moon if he had.

"I really would have loved to have met him one day. I have absolutely no idea what I would say to him if I met him in person, but at the same time I respect that it's his right not to respond."

Since treatment, Sam has seen a complete transformation of health.

Although his gratitude to the anonymous man who saved him was never reciprocated, he lives on with the legacy of his donated stem cells that gave him another chance at life.

"To the best of my knowledge he was the only suitable match for me anywhere in the world, if he hadn't donated at the time there would have been no other alternative for me.

"Almost certainly if I had not received the transplant at the time, almost certainly it would have resulted in my death to be honest."

The future is now looking bright for the graduate.

Sam said: "Everything is going pretty well, the blood donation I needed the transplant for has been completely cured.

"I managed to get out of hospital to do my exams and thankfully I was able to graduate before the end of the year in July 2017.

"It's been completely life changing. In some ways its changed my life much more for the positive.

"If I had not had the transplant when I did, there's no doubt I would not be alive today or my quality of life would be dreadful.

"I can get out and about more, socialise with my friends, I am able to do things that I wouldn't be able to do for quite a while because of my health.

"I feel like my life is starting to return to normal now two years on."

Go here to read the rest:
'My shock cancer scare and the anonymous German who saved me' - Birmingham Live

UK animal experiment statistics indicate reluctance to embrace modern tools to advance British labs into the 21st century – Labmate Online

By Induced Pluripotent Stem Cells

Recently published Home Office statistics [1], have revealed that a high number of dogs, mice, cats, rabbits and other animals some 3.52 million - are still being used in British laboratories despite availability of high-tech and often more human-predictive non-animal approaches.

Humane Society International (HSI) Senior Scientist Dr Lindsay Marshall, who for 12 years managed a laboratory dedicated to animal-free research into respiratory diseases, offers views on some of the reasons that might be preventing a much higher uptake of non-animal techniques that could offer a viable alternative to the use of animal models in research and industry.As a scientist myself, I know all too well the drawbacks of relying on animals to study and treat human disease. The fact is that animal models fail far more often than they succeed, so its hugely frustrating and worrying to see the UK, year after year, failing to move away from outdated animal experiments. Its high time UK research funding bodies stopped squandering British taxpayer money and charitable donations on dead-end research and made a serious investment in human organoids, organs-on-a-chip, computerised systems biology models and other advanced, non-animal technologies that are the true future of modern medical research.Dr Marshall pointed out that in 2010 the Government made a commitment to reduce animals used in scientific research; but almost 10 years after this declaration of intent [2], the UK remains one of the highest lab animal users in Europe. In those same years, non-animal technologies that can produce faster, cheaper and more human-relevant results, have advanced enormously: Computers are much better than animals at predicting possible toxic effects of chemicals and drugs [3] The discovery of induced pluripotent stem cells has helped to remove the ethical barriers to stem cell use [4] Scientists have created human-mimetic systems of almost every organ in the body. There is a human-on-a-chip for drug testing [5], a patient-on-a-chip is not far away [6] and chips have travelled to space to investigate the impact of ageing on the human body [7].

Dr Marshall is not alone in her opinion. A raft of academic reviews from expert scientists in a range of fields reach the same conclusion for conditions as diverse as autism, cardiovascular disease, liver disease, diabetes, and Alzheimers disease [8] and they call for more investment in human-relevant methods.I think that there are many reasons for a reluctance to move away from animals. There is a culture of inertia in research, where animal models have been developed in and are used by a lab, these will be favoured by the researchers and they may see no reason to change or adapt to more relevant, human-focused approaches. Fear of the unknown. Theres an element of familiarity in how to use the animals and understanding the outputs from the animals, that enables persistence of animal models and does not take into account the huge species gap that exists between animals and humans that impacts translation efficiency. Anecdotally, we have heard of more junior researchers being taught and expected to use, the animal methods by their PI, despite a desire to use more human and humane approaches. There is also a requirement to consider the research question in applying non-animal approaches for the first time - there are no simple like-for-like replacements such that a single in vitro assay will stand in for an animal model. We (HSI) do not see this as a reason to continue using the animal models, or to spend scant research resources tweaking existing animal models to create something symptomatically similar to a human condition. Instead, we suggest that researchers look to articulate their research questions in a manner that reflects the novel methodologies emerging and that considers how these methods may be incorporated into a program of research in order to address a specific research question. We believe that framing the question to enable exploitation of the suite of continually developing non-animal methods that are rapidly advancing human relevant science, without compromising safety or discovery, is more likely to translate to much needed treatments and interventions, enables better understanding of human disease. Education and training are required (see below), but at all levels, not just newly qualified researchers.Is the UK government investing enough in research structure support, funding, partnership incentives, graduate/technician education and training?There are some initiatives already- eg the NC3Rs, the Medicines Discovery Catapult - but much of the funding for purely non-animal research is through charities and so is extremely limited and incredibly competitive. The NC3Rs funding for Replacement is combined with initiatives to refine and reduce animal use and it is apparent that this, estimated as around 3% of total research funding in the UK, is not sufficient to encourage the move away from animals. We feel that the Research Councils could use their strategic science roadmaps to help the transitioning of UK life science research to a human biology-based, non-animal paradigm (akin to the US National Academies vision of Toxicity testing in the 21st century or Tox21), with augmented funding for cutting-edge human-relevant technologies and approaches such as human organoids, organs-on-a-chip and elucidation of pathways of human disease and disorders.Education is an important point - there are recognised gaps in training not just for non-animal methods, but ethics and welfare, (see https://www.theguardian.com/higher-education-network/2016/dec/10/we-are-getting-animal-research-wrong-only-education-can-fix-it?CMP=share_btn_tw). Creating a UK workforce that understands the value and utility of non-animal approaches necessitates revising educational curricula to include modern, relevant, non-animal technologies (e.g. human pathways-based methods). Synchronising educational curricula with high level research objectives is needed to develop a strong, capable workforceappropriately qualified researchers responsive to challenges facing UK science (and consistent with implementation of the Animal (Scientific Procedures) Act). The European Union has just announced their intention to develop online modules for training in non-animal approaches (https://iivs.org/2019/07/16/iivs-partners-win-contract-from-ec-for-training-non-animal-testing-methods/), but we obviously do not know of the UKs ability to access this after October. Raising awareness is key, but has to go beyond educating budding scientists and tackle that inertia of the established researchers refusing to put down their mice!What incentives do you feel could be more helpful to industry for example, financial (eg through regulatory changes; tax breaks; employment assistance schemes) and through supported research partnership initiatives?Actually, industry are leading the way- in the UK, the number of animals used for regulatory purposes is on the decrease, as the non-animal methods seem to be embraced by industry, perhaps due to the Tox21 initiative which was developed in the US and uses human cell-based assays to develop more efficient approaches in predicting how substances impact human health. Increases in data outputs and efficiency with these approaches have vastly reduced the use of animals in toxicity testing such that only 26% of procedures in the UK in 2018 were for regulatory purposes. One of the barriers to wider uptake of the non-animal approaches for regulatory purposes are the geographical differences in requirements and we at HSI have been calling for global harmonisation for regulatory requirement for some time, and through our work with intergovernmental bodies like the OECD, we are trying to accelerate global adoption of non-animal testing methods.Recently, reviews of the need for animal research facilities in the UK have led to closure of the Wellcome Sanger Institute [9] illustrating the growing recognition within the scientific community that a paradigm shift away from animal use is essential for medical progress. Recognition that fewer animals are required due to a rise in the use of alternative technologies [10] is a step in the right direction, yet the Home Office animal use statistics indicate that there is much more work required to reduce the body count Dr Marshall added.

1. 2018 Home Office statistics: https://assets.publishing.service.gov.uk/government/uploads/system/uploads/attachment_data/file/818158/annual-statistics-scientific-procedures-living-animals-2018.pdf 2. https://publications.parliament.uk/pa/cm201011/cmhansrd/cm110718/wmstext/110718m0001.htm3. Passini, et al. 2017 Human In Silico Drug Trials Demonstrate Higher Accuracy than Animal Models in Predicting Clinical Pro-Arrhythmic Cardiotoxicity. Front Physiol.8:668.Luechtefeld et al. 2018 Machine learning of toxicological big data enables read-across structure activity relationships (RASAR) outperforming animal test reproducibility. Toxicological Sciences. 165 1, 1 September 2018: 198-2124. https://www.eurostemcell.org/ips-cells-and-reprogramming-turn-any-cell-body-stem-cell5. https://hesperosinc.com6. Edington et al. (2018) Interconnected Microphysiological Systems for Quantitative Biology and Pharmacology Studies. Sci Rep. 2018 Mar 14;8(1):4530. doi: 10.1038/s41598-018-22749-07. https://ncats.nih.gov/tissuechip/projects/space8. Savoji, et al. 2018 Cardiovascular Disease Models: A Game Changing Paradigm in Drug Discovery and Screening. Biomaterials. 10.1016/j.biomaterials.2018.09.036 Boeckmans et al. 2018. Human-based systems: Mechanistic NASH modelling just around the corner? Pharmacol Res. 134:257-267. 10.1016/j.phrs.2018.06.029 Muotri, A. R. 2016. The Human Model: Changing Focus on Autism Research. Biol Psychiatry. 79;8: 642-9. Bowman, et al. 2018. Future Roadmaps for Precision Medicine Applied to Diabetes: Rising to the Challenge of Heterogeneity. Journal of Diabetes Research. 10.1155/2018/3061620 Clerc, et al. 2016. A look into the future of ALS research. Drug Discov Today. 21;6: 939-499. https://www.sanger.ac.uk/news/view/sanger-institute-animal-research-facility-closehttps://www.nature.com/articles/d41586-019-02002-y10. https://chemicalwatch.com/77872/sanger-institute-announces-closure-of-animal-research-facility11. https://ntp.niehs.nih.gov/iccvam/meetings/iccvam-forum-2019/06-lee-ncats_508.pdf

Are you working with methods or ideas that could transform the need for animals in research? Are you a producer of technology that reduces the needs for such tests?We would welcome your feedback on the above please email heather@intlabmate.com

Articles are free to download. Please login to read this article or create an account.

See the original post here:
UK animal experiment statistics indicate reluctance to embrace modern tools to advance British labs into the 21st century - Labmate Online

Sangamo Announces Gene Therapy and Ex Vivo Gene-Edited Cell Therapy Data Presentations at the American Society of Hematology Annual Meeting – Yahoo…

By Stem Cell Medicine

BRISBANE, Calif.--(BUSINESS WIRE)--

Sangamo Therapeutics, Inc. (SGMO), a genomic medicine company, today announced that hemophilia A gene therapy clinical data and hemoglobinopathies ex vivo gene-edited cell therapy data will be featured in poster presentations at the 61st Annual Meeting of the American Society of Hematology (ASH). The ASH abstracts, which were submitted on August 3, 2019, were released online this morning. The conference will take place in Orlando, FL, from December 7-10, 2019.

Gene Therapy

The SB-525 poster will show updated Alta study data including durability of Factor VIII (FVIII) levels, bleeding rate, factor usage, and safety, for all five patients in the high dose cohort of 3e13 vg/kg, with approximately 4 months to 11 months of follow-up after treatment with SB-525.

As of the abstract submission date, four patients in the 3e13 vg/kg cohort achieved FVIII levels within the normal range with no bleeding events reported up to 24 weeks post-administration. These patients did not require FVIII replacement therapy following the initial prophylactic period of up to approximately 3 weeks post-SB-525 administration. The fifth patient in the 3e13 vg/kg cohort had only recently undergone treatment with SB-525 at the time of the abstract submission. As previously reported, one patient had treatment-related serious adverse events (SAEs) of hypotension and fever, which occurred approximately 6 hours after completion of the vector infusion and resolved with treatment within 24 hours, with no loss of FVIII expression. SB-525 is being developed as part of a global collaboration between Sangamo and Pfizer.

The rapid kinetics of Factor VIII expression, durability of response, and the relatively low intra-cohort variability in the context of a complete cessation of bleeding events and elimination of exogenous Factor VIII usage continues to suggest SB-525 is a differentiated hemophilia A gene therapy, said Bettina Cockroft, M.D., M.B.A., Chief Medical Officer of Sangamo, commenting on the published abstract. We are pleased with the progress of the program toward a registrational Phase 3 study led by Pfizer, who announced it has enrolled its first patient in the 6-month Phase 3 lead-in study. We have recently completed the manufacturing technology transfer to Pfizer and initiated the transfer of the IND.

Ex Vivo Gene-Edited Cell Therapy

The ST-400 beta thalassemia poster will show preliminary results from the first three patients enrolled in the Phase 1/2 THALES study. In this study, hematopoietic stem progenitor cells (HSPCs) are apheresed from the patient, edited to knock out the erythroid specific enhancer of the BCL11A gene, and cryopreserved prior to infusion back into the patient following myeloablative conditioning with busulfan. The first three patients all have severe beta thalassemia genotypes: 0/0, homozygous for the severe + IVS-I-5 (G>C) mutation, and 0/+ genotype including the severe IVS-II-654 (C>T) mutation, respectively.

As of the abstract submission date, Patient 1 and Patient 2 had experienced prompt hematopoietic reconstitution. Patient 1 had increasing fetal hemoglobin (HbF) fraction that contributed to a stable total hemoglobin. After being free from packed red blood cell (PRBC) transfusions for 6 weeks, the patient subsequently required intermittent transfusions. Patient 2 had rising HbF levels observed through 90 days post-infusion. For both patients, as of the most recent follow-up reported in the abstract, on-target insertions and deletions (indels) were present in circulating white blood cells. Patient 3 had just completed ST-400 manufacturing at the time of abstract submission. As previously disclosed, Patient 1 experienced an SAE of hypersensitivity during ST-400 infusion considered by the investigator to be related to the product cryoprotectant, DSMO, and which resolved by the end of the infusion. No other SAEs related to ST-400 have been reported and all other AEs have been consistent with myeloablation. No clonal hematopoiesis has been observed. Longer follow-up will be required to assess the clinical significance of these early results. ST-400 is being developed as part of a global collaboration between Sangamo and Sanofi, along with support through a grant from the California Institute for Regenerative Medicine (CIRM).

Story continues

The first three patients enrolled in the THALES study all have severe beta thalassemia genotypes that result in almost no endogenous beta globin production. The increases in fetal hemoglobin and presence of on-target indels in circulating blood cells suggests successful editing using zinc finger nucleases. The results are preliminary and will require additional patients and longer-term follow-up to assess their clinical significance, said Adrian Woolfson, BM., B.Ch., Ph.D., Head of Research and Development. It is important to note that myeloablative hematopoietic stem cell transplantation reboots the hematopoietic system, and that sufficient time is required for the stem cells to fully repopulate the marrow and for new blood cells to form. In other myeloablative conditioning studies in a similar patient population, full manifestation of the effects of gene modification in the red blood cell compartment has taken as long as 12 months or more to become evident.

Sanofis in vitro sickle cell disease poster details a similar approach to ST-400, using mobilized HSPCs from normal donors and SCD patients and utilizing the same zinc finger nuclease for gene editing, delivered as transient non-viral RNA, and designed to disrupt the erythroid specific enhancer of the BCL11A gene, which represses the expression of the gamma globin genes, thereby switching off HbF synthesis. Results from ex vivo studies demonstrated enriched biallelic editing, increased HbF, and reduced sickling in erythroid cells derived from non-treated sickle cell disease patients. Sanofi has initiated a Phase 1/2 trial evaluating BIVV003, an ex vivo gene-edited cell therapy using ZFN gene editing technology to modify autologous hematopoietic stem cells using fetal hemoglobin to produce functional red blood cells with higher BhF content that are resistant to sickling in patients with severe sickle cell disease. Recruitment is ongoing.

About the Alta study

The Phase 1/2 Alta study is an open-label, dose-ranging clinical trial designed to assess the safety and tolerability of SB-525 gene therapy in patients with severe hemophilia A. SB-525 was administered to 11 patients in 4 cohorts of 2 patients each across 4 ascending doses (9e11 vg/kg, 2e12 vg/kg, 1e13vg/kg and 3e13vg/kg) with expansion of the highest dose cohort by 3 additional patients. The U.S. Food and Drug Administration (FDA) has granted Orphan Drug, Fast Track, and regenerative medicine advanced therapy (RMAT) designations to SB-525, which also received Orphan Medicinal Product designation from the European Medicines Agency.

About the THALES study

The Phase 1/2 THALES study is a single-arm, multi-site study to assess the safety, tolerability, and efficacy of ST-400 autologous hematopoietic stem cell transplant in 6 patients with transfusion-dependent beta thalassemia (TDT). ST-400 is manufactured by ex vivo gene editing of a patient's own (autologous) hematopoietic stem cells using non-viral delivery of zinc finger nuclease technology. The THALES study inclusion criteria include all patients with TDT (0/0 or non- 0/0) who have received at least 8 packed red blood cell transfusions per year for the two years before enrollment in the study. The FDA has granted Orphan Drug status to ST-400.

About Sangamo Therapeutics

Sangamo Therapeutics, Inc. is focused on translating ground-breaking science into genomic medicines with the potential to transform patients' lives using gene therapy, ex vivo gene-edited cell therapy, in vivo genome editing, and gene regulation. For more information about Sangamo, visit http://www.sangamo.com.

Forward-Looking Statements

This press release contains forward-looking statements regarding Sangamo's current expectations. These forward-looking statements include, without limitation, statements regarding the Company's ability to develop and commercialize product candidates to address genetic diseases with the Company's proprietary technologies, as well as the timing of commencement of clinical programs and the anticipated benefits therefrom. These statements are not guarantees of future performance and are subject to certain risks, uncertainties and assumptions that are difficult to predict. Factors that could cause actual results to differ include, but are not limited to, the outcomes of clinical trials, the uncertain regulatory approval process, uncertainties related to the execution of clinical trials, Sangamo's reliance on partners and other third-parties to meet their clinical and manufacturing obligations, and the ability to maintain strategic partnerships. Further, there can be no assurance that the necessary regulatory approvals will be obtained or that Sangamo and its partners will be able to develop commercially viable product candidates. Actual results may differ from those projected in forward-looking statements due to risks and uncertainties that exist in Sangamo's operations and business environments. These risks and uncertainties are described more fully in Sangamo's Annual Report on Form 10-K for the year ended December 31, 2018 as filed with the Securities and Exchange Commission and Sangamo's most recent Quarterly Report on Form 10-Q. Forward-looking statements contained in this announcement are made as of this date, and Sangamo undertakes no duty to update such information except as required under applicable law.

View source version on businesswire.com: https://www.businesswire.com/news/home/20191106005273/en/

Read the original post:
Sangamo Announces Gene Therapy and Ex Vivo Gene-Edited Cell Therapy Data Presentations at the American Society of Hematology Annual Meeting - Yahoo...

Vertex invests in gene therapy manufacturing – BioPharma-Reporter.com

By Stem Cell Clinic

Across 2019, Vertex has struck deals intended to yield a new generation of breakthrough medicines.

In June, Vertex agreed to pay $245m (220m) upfront to acquire Exonics Therapeutics for its gene editing technology and pipeline of programs targeting diseases including Duchenne muscular dystrophy (DMD). Months later, Vertex put up another $950m to buy Semma Therapeutics and its cell therapy treatment for type I diabetes.

The acquisitions moved Vertex, which started out in small molecules, into new areas, and building out capabilities in those areas will cost money.

In recent years, Vertex has grown its annual operating expenses by 10% to 14%. Talking on a recent quarterly results conference call, Vertex CFO Charles Wagner warned investors to expect costs to rise faster in 2020.

Wagner said, Our current expectation is that the rate of growth will be somewhat higher in 2020 as we invest in research and preclinical manufacturing for selling genetic therapies in support of our programs in type I diabetes, DMD and other diseases.

The move into type I diabetes also takes Vertex into territory that, to some observers, looks different than the areas the company has targeted historically.

Asked by an analyst about the shift in focus, Vertex CEO Jeff Leiden downplayed the differences, noting that type I diabetes is treated in the US in a relatively small number ofcenters that can be targeted by a speciality sales force.

Researchers have achieved positive, long-term outcomes by transplanting cadaveric islets into patients but two barriers have stopped companies from industrialising that approach.

Firstly, there are too few cadaveric islets to treat all type I diabetics. Secondly, immunosuppression is needed to stop patients from rejecting the transplanted cells.

Semma is trying to tackle the problems by differentiating stem cells and using a device to protect them from the immune system. Vertex thinks these technologies are the breakthroughs the field needs to industrialize the concept.

Leiden said, We were watching companies who are addressing those two problems for the last two, three years. And over the last six to eight months, we were convinced that Semma has actually solved both of those problems.

Vertex reached that conclusion on the strength of preclinical data. Now, Vertex is set to invest to find out whether the idea works in the clinic.

Read the original:
Vertex invests in gene therapy manufacturing - BioPharma-Reporter.com

[Weekly Funding Roundup] Startups raised $53.3M, a drop of 75.8 pc from last week – YourStory

By Stem Cell Clinic

This week, startups raised $53.3 million in equity funding, down 75.8 percent as compared to last week's $220.7 million. Three venture debt deals were also signed that saw startups raising a total of $6.1 million.

Weekly Funding Roundup

PayU co-founder Jitendra Guptas latest venture, Digifin raised $24 million in an early-stage round from Matrix Partners, Sequoia Capital, Greyhound Capital, 3One4 Capital, Rocket Internet, BeeNext, and Tanglin Venture Partners. The company operates under Amica Financial Technologies.

Exam preparation startup Gradeup this week said it had received $7 million in Series A funding from Times Internet. This is the second fundraise for Gradeup, taking the total funds raised by the startup to $10 million. Gradeup will use the funds to enhance its technology and product capabilities, diversify offerings, and scale its academic team.

Online learning platform Adda247 raised Series B funding of $6 million led by Infoedge (India) Ltd and Asha Impact. STL, a current investor of Adda247, also participated in the latest round. With this round, Adda247 has raised a total of $10 million of funds to date.

Delhi-based healthcare platform BeYouPlus raised$3.2 millionin its Series A funding round, led byIvyCap Ventures.The round also saw participation fromMadison CapitalandSingapore Angel Network, among others.

SafeHouse Technologies, that focusses on mobile-first cybersecurity, raised $2.2 million in a seed funding round led by Barclays UK Ventures, the venture capital business unit of British multinational financial services group Barclays.

Residential proptech startup YourOwnROOM raised $1.3 million in seed fundingfrom a group of investors that includes US-based investment firm Lotus Capital; Ravi Chaturvedi, former President of P&G; Narasimha Murthy, Co-founder of a US-based healthtech company; and a group of angel investors based out of the USand Bengaluru.

Bengaluru-based transport service for kids, PiggyRide raised $1.05 million (Rs 7.5 crore) led by JAFCO Investment Asia Pacific, along with a clutch of angel investors, including InMobis Naveen Tewari; Livspace Co-founders Ramakant Sharma and Anuj Srivastava; Goldman Sachs' MD, Niladri Mukhopadhyay; Zipdials Amiya Pathak; and EzCreds Sachin Maheshwari.

Pet-care platform Wiggles raised angel funding of $1 million from investors such asNachikhet Deshpande, COO of L&T Infotech; Aparna Badkundri, Director, Dell Computers; Sachin Phadke, MD of Vetbiochem India; Abhay Amrute, Senior Partner, IIFL Wealth Management Ltd; Satish Billakota, VP, Europe Cognizant; Risshee Tandulwadkar, Founder, Solo Stem Cell Clinic, apart from a few other HNIs.

Mumbai-based edtech startup ENpower, which provides entrepreneurship learning programmes for teenagers, raised seed funding of $0.37 million (Rs 2.65 crore) from an investor consortium led by Nikhil Vora, Founder & CEO of Sixth Sense Ventures, a consumer-centric venture fund.

The Knotty Tales, a tech startup that acts as a digital wedding planning platform, raised $0.06 million from a group of angel investors as a part of its seed round. This fundraising will be used to focus on new product innovation and further strengthening relationships with clients and vendors.

Seed investor Venture Catalysts invested an undisclosed amount in deep-tech computer vision and augmented reality (AR) specific startup peAR Technologies.

Mitrata Financial Services, a women-focussed microlender, raised Series A funding from a group of individual investors. Sadaf Sayeed, CEO of Muthoot Microfin, was one of the investors in Mitrata.

Mumbai-based podcast startup Kuku FM raised an undisclosed amount from the dream team of VCs 3one4 Capital, Shunwei Capital (a Chinese investment firm spun off from handset-maker Xiaomi), and India Quotient. This investment has raised the bar for the Kuku FM team. The funds raised will be used to expand its content library and increase its user base.

Vehicle maintenance startup Hoopy raised an undisclosed amount of funding from Lead Angels and Venture Farmer. Bengaluru-based family office Venture Farmer typically makes Pre-Series A to Series B investments ranging between $100k-$700k.

Delhi-based startup goStops, a premium chain of youth traveller hostels, raised an undisclosed amount of funds in its initial funding round from angel investors.

Corefactors, an integrated sales and marketing company, raised an undisclosed amount in its seed round from Mumbai based angel investment firm, Ah! Ventures.

There was one late-stage deal this week. Omnichannel furniture brand Urban Ladder has raised close to $2.1 million (Rs 14.91 crore) from SAIF Partners, Steadview Capital and Sequoia Capital India, as a follow-on of its Series E round, according to regulatory filings accessed by YourStory.

News and classifieds platform Lokal raised $3 million from 3one4 Capital, Y Combinator, RB Investments, SOMA Capital, and its existing investor India Quotient. Focused on regional languages, Lokal will be using the freshly raised funds to improve its product and expand to newer geographies.

Data, analytics, and decisioning company Experian has picked up a strategic stake in smart data mobile marketing platform Vserv Digital Services. While the deal size and amount were undisclosed, the company stated the investment was in line with its vision to boost financial inclusion, by ensuring a friction-free digital onboarding experience for its consumers.

Hyderabad-based Thinkwide PGO raised $1 million in equity funding from Sreeni Musani of Ektha Pvt. Ltd. and Narsi Reddy Posham of IRA Reality Pvt. Ltd. The startup has also received an additional commitment of $1 million in equity investment from the same set of investors.

Online furniture rental firm Furlenco raised Rs 2.5 crore ($1.6 million) debt funding from Sandeep Baid, Shakuntalam Holdings, and Ritona Vincom.The company has issued 250 non-convertible debentures (NCDs) of face value Rs 1 lakh each for the same.

Consumer and small and medium enterprises (SMEs) focussed digital lending app InCred raised Rs 31.4 crore ($4.4 million) in its second round of debt financing.

Furniture and electronics leasing platform, Rentomojo raised Rs 1 crore ($0.14 million) as non-convertible debentures (NCDs) from Kamal Bhandari on private placement basis.

Used vehicle marketplace Droom acquired NBFC Xeraphin Finvest Pvt. Ltd.

SoftBank-backed robotic process automation (RPA) player Automation Anywhere which acqui-hired engineering startup Cathyos Labs.

And finally, Netcore Solutions acquiredAI startup Boxx.ai.

(Edited by Suman Singh and Saheli Sen Gupta)

Original post:
[Weekly Funding Roundup] Startups raised $53.3M, a drop of 75.8 pc from last week - YourStory

→←